COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has…
Articles by Larry Luxner
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested…
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500…
NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts
As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise that people with rare diseases —…
NORD’s Advice on COVID-19: You’re Stronger Than You Think
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably…
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains…
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business…
HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and…
NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a…
At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo…